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    Clinical Trial Results:
    A Randomized, Open-label Eculizumab and Ravulizumab Controlled, Non-Inferiority Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria who are Currently Treated with Eculizumab or Ravulizumab

    Summary
    EudraCT number
    		 2020-002761-33
    Trial protocol
    		 IT   DE   ES   FR   PL   GR  
    Global end of trial date
    12 Jul 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    25 Jul 2024
    First version publication date
    25 Jul 2024
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    R3918-PNH-2022
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05131204
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Regeneron Pharmaceuticals, Inc.
    Sponsor organisation address
    777 Old Saw Mill River Road, Tarrytown, NY, United States, 10591
    Public contact
    Clinical Trials Administrator, Regeneron Pharmaceuticals, Inc., 001 844-734-6643, clinicaltrials@regeneron.com
    Scientific contact
    Clinical Trials Administrator, Regeneron Pharmaceuticals, Inc., 001 844-734-6643, clinicaltrials@regeneron.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    12 Jul 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    12 Jul 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of the study was: To evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in participants with PNH who switch from eculizumab or ravulizumab therapy versus participants who continue their eculizumab or ravulizumab therapy.
    Protection of trial subjects
    It is the responsibility of both the sponsor and the investigator(s) to ensure that this clinical study will be conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki, and that are consistent with the ICH guidelines for GCP and applicable regulatory requirements.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    06 Oct 2022
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Greece: 1
    Country: Number of subjects enrolled
    Korea, Republic of: 1
    Country: Number of subjects enrolled
    Philippines: 1
    Worldwide total number of subjects
    3
    EEA total number of subjects
    1
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    3
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 A total of 140 participants were expected to be enrolled, however, due to feasibility: five participants were screened and 3 were randomized and treated. There were 2 screen failures.

    Period 1
    Period 1 title
    Overall study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Pozelimab and Cemdisiran
    Arm description
    		 Randomized 1:1
    Arm type
    		 Experimental

    Investigational medicinal product name
    Pozelimab
    Investigational medicinal product code
    R3918
    Other name
    Pharmaceutical forms
    Cutaneous liquid
    Routes of administration
    Intravenous use
    Dosage and administration details
    Administered per protocol

    Investigational medicinal product name
    Ravulizumab
    Investigational medicinal product code
    ALXN1210
    Other name
    Ultomiris
    Pharmaceutical forms
    Cutaneous liquid
    Routes of administration
    Intravenous use
    Dosage and administration details
    Administered per protocol

    Investigational medicinal product name
    Eculizumab
    Investigational medicinal product code
    Other name
    Soliris
    Pharmaceutical forms
    Cutaneous liquid
    Routes of administration
    Intravenous use
    Dosage and administration details
    Administered per protocol

    Investigational medicinal product name
    Cemdisiran
    Investigational medicinal product code
    ALN-CC5
    Other name
    Pharmaceutical forms
    Cutaneous liquid
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Administered per protocol

    Arm title
    		 Anti-C5 standard-of-care
    Arm description
    		 Randomized 1:1
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ravulizumab
    Investigational medicinal product code
    ALXN1210
    Other name
    Ultomiris
    Pharmaceutical forms
    Cutaneous liquid
    Routes of administration
    Intravenous use
    Dosage and administration details
    Administered per protocol

    Investigational medicinal product name
    Eculizumab
    Investigational medicinal product code
    Other name
    Soliris
    Pharmaceutical forms
    Cutaneous liquid
    Routes of administration
    Intravenous use
    Dosage and administration details
    Administered per protocol

    Number of subjects in period 1
    		Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Started
    2
    1
    Completed
    0
    0
    Not completed
    2
    1
         Sponsor decision
    2
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Pozelimab and Cemdisiran
    Reporting group description
    		 Randomized 1:1

    Reporting group title
    Anti-C5 standard-of-care
    Reporting group description
    		 Randomized 1:1

    Reporting group values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care Total
    Number of subjects
    		 2 1 3
    Age Categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 2 1 3
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Gender Categorical
    Units: Subjects
        Female
    		 1 0 1
        Male
    		 1 1 2
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0
        Asian
    		 1 1 2
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Black or African American
    		 0 0 0
        White
    		 1 0 1
        More than one race
    		 0 0 0
        Unknown or Not Reported
    		 0 0 0
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 0 0 0
        Not Hispanic or Latino
    		 2 1 3
        Unknown or Not Reported
    		 0 0 0

    End points

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    End points reporting groups
    Reporting group title
    Pozelimab and Cemdisiran
    Reporting group description
    		 Randomized 1:1

    Reporting group title
    Anti-C5 standard-of-care
    Reporting group description
    		 Randomized 1:1

    Primary: Percent change in lactate dehydrogenase (LDH) from baseline to week 36

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    End point title
    		 Percent change in lactate dehydrogenase (LDH) from baseline to week 36 [1]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline to week 36
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No data was collected so no statistical analysis was completed.
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [2]
    0 [3]
    Units: Percentage of change
        number (not applicable)
    Notes
    [2] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [3] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Percentage of participants with transfusion avoidance after day 1 through week 36

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    End point title
    		 Percentage of participants with transfusion avoidance after day 1 through week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Day 1 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [4]
    0 [5]
    Units: Percentage
        number (not applicable)
    Notes
    [4] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [5] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Percentage of participants with transfusion avoidance from week 4 through week 36

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    End point title
    		 Percentage of participants with transfusion avoidance from week 4 through week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Week 4 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [6]
    0 [7]
    Units: Percentage
        number (not applicable)
    Notes
    [6] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [7] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Percentage of participants with breakthrough hemolysis after day 1 through week 36

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    End point title
    		 Percentage of participants with breakthrough hemolysis after day 1 through week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Day 1 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [8]
    0 [9]
    Units: Percentage
    Notes
    [8] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [9] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Percentage of participants with hemoglobin stabilization after day 1 through week 36

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    End point title
    		 Percentage of participants with hemoglobin stabilization after day 1 through week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Day 1 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [10]
    0 [11]
    Units: Percentage
    Notes
    [10] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [11] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Percentage of participants with breakthrough hemolysis from week 4 through week 36

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    End point title
    		 Percentage of participants with breakthrough hemolysis from week 4 through week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Week 4 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [12]
    0 [13]
    Units: Percentage
    Notes
    [12] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [13] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Percentage of participants with hemoglobin stabilization from week 4 through week 36

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    End point title
    		 Percentage of participants with hemoglobin stabilization from week 4 through week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Week 4 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [14]
    0 [15]
    Units: Percentage
    Notes
    [14] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [15] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Percentage of participants who maintained adequate control of hemolysis from week 8 through week 36

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    End point title
    		 Percentage of participants who maintained adequate control of hemolysis from week 8 through week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Week 8 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [16]
    0 [17]
    Units: Percentage of participants
    Notes
    [16] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [17] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Percentage of participants with adequate control of LDH after day 1 though week 36

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    End point title
    		 Percentage of participants with adequate control of LDH after day 1 though week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Day 1 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [18]
    0 [19]
    Units: Percentage
    Notes
    [18] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [19] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Percentage of participants with adequate control of LDH from week 8 through week 36

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    End point title
    		 Percentage of participants with adequate control of LDH from week 8 through week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Week 8 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [20]
    0 [21]
    Units: Percentage
    Notes
    [20] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [21] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Percentage of participants with normalization of LDH after day 1 through week 36

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    End point title
    		 Percentage of participants with normalization of LDH after day 1 through week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Day 1 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [22]
    0 [23]
    Units: Percentage
    Notes
    [22] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [23] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Percentage of participants with normalization of LDH from week 8 through week 36

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    End point title
    		 Percentage of participants with normalization of LDH from week 8 through week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Week 8 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [24]
    0 [25]
    Units: Percentage
    Notes
    [24] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [25] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Percentage of participants who maintained adequate control of hemolysis after day 1 through week 36

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    End point title
    		 Percentage of participants who maintained adequate control of hemolysis after day 1 through week 36
    End point description
    End point type
    Secondary
    End point timeframe
    After day 1 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [26]
    0 [27]
    Units: Percentage of participants
    Notes
    [26] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [27] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Change in fatigue as measured by the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale from Baseline to Week 36

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    End point title
    		 Change in fatigue as measured by the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale from Baseline to Week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [28]
    0 [29]
    Units: Score on a scale
    Notes
    [28] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [29] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Change in global health status (GHS)/QoL scale score on the EORTC-QLQ-C30 from baseline to week 36

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    End point title
    		 Change in global health status (GHS)/QoL scale score on the EORTC-QLQ-C30 from baseline to week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [30]
    0 [31]
    Units: Score on a scale
    Notes
    [30] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [31] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Change in Physical Function (PF) score on the European organization for research and treatment of cancer quality-of-Life questionnaire Core 30 Items (EORTC-QLQ-C30) from baseline to week 36

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    End point title
    		 Change in Physical Function (PF) score on the European organization for research and treatment of cancer quality-of-Life questionnaire Core 30 Items (EORTC-QLQ-C30) from baseline to week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [32]
    0 [33]
    Units: Score on a scale
    Notes
    [32] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [33] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Rate of RBCs transfused per protocol algorithm after day 1 through week 36

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    End point title
    		 Rate of RBCs transfused per protocol algorithm after day 1 through week 36
    End point description
    End point type
    Secondary
    End point timeframe
    After day 1 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [34]
    0 [35]
    Units: Rate of transfusion
    Notes
    [34] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [35] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Rate of RBCs transfused per protocol algorithm from week 4 through week 36

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    End point title
    		 Rate of RBCs transfused per protocol algorithm from week 4 through week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Week 4 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [36]
    0 [37]
    Units: Rate of transfusion
    Notes
    [36] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [37] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Number of units of RBCs transfused per protocol algorithm after day 1 through week 36

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    End point title
    		 Number of units of RBCs transfused per protocol algorithm after day 1 through week 36
    End point description
    End point type
    Secondary
    End point timeframe
    After day 1 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [38]
    0 [39]
    Units: Number
    Notes
    [38] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [39] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Number of units of RBCs transfused per protocol algorithm from week 4 through week 36

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    End point title
    		 Number of units of RBCs transfused per protocol algorithm from week 4 through week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Week 4 through week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [40]
    0 [41]
    Units: Number
    Notes
    [40] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [41] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Change in hemoglobin levels from baseline to week 36

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    End point title
    		 Change in hemoglobin levels from baseline to week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [42]
    0 [43]
    Units: Change
    Notes
    [42] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [43] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Incidence and severity of treatment emergent serious adverse events (SAEs) over 36 weeks

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    End point title
    		 Incidence and severity of treatment emergent serious adverse events (SAEs) over 36 weeks
    End point description
    End point type
    Secondary
    End point timeframe
    Over 36 weeks
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [44]
    0 [45]
    Units: Events
    Notes
    [44] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [45] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Incidence and severity of treatment-emergent adverse events (TEAEs) of special interest over 36 weeks

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    End point title
    		 Incidence and severity of treatment-emergent adverse events (TEAEs) of special interest over 36 weeks
    End point description
    End point type
    Secondary
    End point timeframe
    Over week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [46]
    0 [47]
    Units: Events
    Notes
    [46] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [47] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Incidence and severity TEAEs leading to treatment discontinuation over 36 weeks

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    End point title
    		 Incidence and severity TEAEs leading to treatment discontinuation over 36 weeks
    End point description
    End point type
    Secondary
    End point timeframe
    Over week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [48]
    0 [49]
    Units: Events
    Notes
    [48] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [49] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Change in total CH50 from baseline to week 36

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    End point title
    		 Change in total CH50 from baseline to week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [50]
    0 [51]
    Units: Units per milliliter (U/mL)
    Notes
    [50] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [51] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Concentrations of total cemdisiran in plasma through week 32

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    End point title
    		 Concentrations of total cemdisiran in plasma through week 32
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline through week 32
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [52]
    0 [53]
    Units: Concentration
    Notes
    [52] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [53] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Concentrations of total pozelimab in serum through week 62

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    End point title
    		 Concentrations of total pozelimab in serum through week 62
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline through week 62
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [54]
    0 [55]
    Units: Concentration
    Notes
    [54] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [55] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Concentration of total C5 in plasma through week 62

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    End point title
    		 Concentration of total C5 in plasma through week 62
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to week 62
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [56]
    0 [57]
    Units: Concentration
    Notes
    [56] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [57] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Percent change in total CH50 from baseline to week 36

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    End point title
    		 Percent change in total CH50 from baseline to week 36
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to week 36
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [58]
    0 [59]
    Units: Percentage of change
    Notes
    [58] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [59] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Concentrations of total ravulizumab in plasma through week 44

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    End point title
    		 Concentrations of total ravulizumab in plasma through week 44
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline through week 44
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [60]
    0 [61]
    Units: Concentration
    Notes
    [60] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [61] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Concentrations of total eculizumab in serum through week 40

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    End point title
    		 Concentrations of total eculizumab in serum through week 40
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline through week 40
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [62]
    0 [63]
    Units: Concentration
    Notes
    [62] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [63] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Incidence of treatment emergent anti-drug antibodies (ADAs) to pozelimab through week 62

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    End point title
    		 Incidence of treatment emergent anti-drug antibodies (ADAs) to pozelimab through week 62
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline through week 62
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [64]
    0 [65]
    Units: ADAs
    Notes
    [64] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [65] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Secondary: Incidence of treatment emergent ADAs to cemdisiran through week 62

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    End point title
    		 Incidence of treatment emergent ADAs to cemdisiran through week 62
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline through week 62
    End point values
    		 Pozelimab and Cemdisiran Anti-C5 standard-of-care
    Number of subjects analysed
    0 [66]
    0 [67]
    Units: ADAs
    Notes
    [66] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    [67] - Due to Early Termination, endpoint removed from Statistical Analysis Plan. Data not collected.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From first dose to week 29
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    Pozelimab Q4W and Cemdisiran
    Reporting group description
    		 Randomized 1:1

    Reporting group title
    Anti-C5 standard-of-care
    Reporting group description
    		 Randomized 1:1

    Serious adverse events
    		 Pozelimab Q4W and Cemdisiran Anti-C5 standard-of-care
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 1 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Pozelimab Q4W and Cemdisiran Anti-C5 standard-of-care
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 2 (100.00%)
    1 / 1 (100.00%)
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1
    Blood glucose increased
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    General disorders and administration site conditions
    Injection site rash
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Skin and subcutaneous tissue disorders
    Eczema
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    21 Jun 2022
    Main purpose for this amendment was to include new secondary endpoints and provide details on the planned analyses.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    20Dec2022 - sponsor decided to terminate study due to feasability challenges related to enrollment. There were no safety or efficacy concerns.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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